a study to evaluate the safety and effectiveness of luspatercept for the treatment of transfusion-dependent (td) anemia associated with myelodysplastic syndromes (mds) & beta-thalassemia (β-thal) in india

Conditions: Anemia

Summary: the purpose of this study is to evaluate the safety and effectiveness of luspatercept in participants who require regular blood cell transfusions due to b-thalassemia and myelodysplastic syndromes in india


Eligibility criteria:
inclusion criteria: β-thalassemia cohort * documented diagnosis of β-thalassemia or hemoglobin (hb e/β-thalassemia). (β-thalassemia with mutation and/or multiplication of alpha \[α\] globin is allowed). * regularly transfused, defined as 6 rbc units to 20 rbc units in the 24 weeks prior to enrollment and no transfusion-free period for \> 35 days during that period. mds-rs cohort - participant has documented diagnosis of mds according to world health organization (who) (2016)/french-american-british fab classification that meets revised international prognostic scoring system (ipss-r) classification of very low, low, or intermediate risk disease and the following criteria: i) rs ≥ 15% of erythroid precursors in bone marrow. if the sf3b1 mutation is present, rs ≥ 5% will be included. ii) less than 5% blasts in bone marrow and \< 1% peripheral blood blasts. iii) peripheral blood white blood cell (wbc) count \< 13,000/ microliters (μl). * if the participant was previously treated with erythropoiesis-stimulating agents (esas) or granulocyte colony-stimulating factor (g-csf)/granulocyte-macrophage colony-stimulating factor (gm-csf), both agents must have been discontinued ≥ 4 weeks prior to the date of enrollment. exclusion criteria: β-thalassemia cohort * a diagnosis of hb s/β-thalassemia or α-thalassemia (for exampe, hemoglobin h). * deep vein thrombosis (dvt) or stroke requiring medical intervention ≤ 24 weeks prior to enrollment. * use of chronic anticoagulant therapy is excluded unless the treatment stopped at least 28 days prior to enrollment. anticoagulant therapies used for prophylaxis for surgery or high-risk procedures as well as low-molecular-weight (lmw) heparin for superficial venous thrombosis and chronic aspirin are allowed. * cytotoxic agents or immunosuppressants or immunomodulatory drugs (imids) ≤ 28 days prior to enrollment (ie, antithymocite globulin or cyclosporine or thalidomide). mds-rs cohort * mds associated with del 5q cytogenetic abnormality. * secondary mds, that is, mds that is known to have arisen as the result of chemical injury or treatment with chemotherapy and/or radiation for other diseases. * participant has known clinically significant anemia due to iron, vitamin b12, or folate deficiencies; autoimmune or hereditary hemolytic anemia; or gastrointestinal bleeding. * iron deficiency to be determined by serum ferritin ≤ 15 micrograms per liter (μg/l) and additional testing if clinically indicated (for example, calculated transferrin saturation \[iron/total iron binding capacity ≤ 20%\] or bone marrow aspirate \[bma\] stain for iron).

Keywords: luspatercept ace-536 transfusion dependent beta-thalassemia myelodysplastic syndromes with ring sideroblasts anemia hemoglobinopathies hematologic diseases bone marrow diseases